In a significant advancement for rare disease treatment in Japan, the Ministry of Health, Labour and Welfare (MHLW) has approved Bylvay® (odevixibat) for the management of pruritus associated with progressive familial intrahepatic cholestasis (PFIC). This decision, reported by The Manilla Times, introduces the first once-daily, oral, ileal bile acid transport inhibitor (IBATi) for PFIC, offering patients, from infants to adults, a non-surgical solution to a devastating condition.
Understanding PFIC and Its Impact
PFIC is a rare genetic liver disorder, affecting an estimated 100 children and infants in Japan. Characterized by abnormal bile acid accumulation in the liver, PFIC leads to progressive liver damage and, if untreated, potentially liver failure. One of the most debilitating symptoms is severe, unrelenting itching (pruritus), which disrupts sleep, impairs cognitive and social development, and significantly diminishes quality of life for both patients and their families.
There are multiple subtypes of PFIC, with types 1 and 2 typically manifesting in early childhood. The disease is equally prevalent among males and females, with onset ranging from infancy to adolescence. Until now, treatment options were limited, often involving invasive surgical interventions.
Bylvay: A New Therapeutic Option
Bylvay acts locally in the small intestine, reducing the reabsorption of bile acids back to the liver and thus lowering their toxic buildup. The drug’s approval in Japan is based on robust evidence from both global and local Phase III clinical trials. In the pivotal PEDFIC study—the largest global Phase III trial in PFIC—children treated with Bylvay experienced significant reductions in pruritus and serum bile acid levels. Importantly, Bylvay was generally well-tolerated, with no serious drug-related adverse events and a low incidence of gastrointestinal side effects.
The Japanese approval followed a local Phase III, open-label study in pediatric patients with PFIC types 1 and 2. This study confirmed the efficacy and safety results observed globally, underpinning Bylvay’s suitability for the Japanese population.
Expert and Patient Perspectives
Medical experts emphasize that early diagnosis and intervention are crucial in PFIC to manage symptoms and preserve liver function. Dr. Hiroki Kondou, Associate Professor at Kindai University Nara Hospital, noted that Bylvay’s approval not only offers symptom relief but may also improve sleep quality and support liver health, greatly benefiting patients and caregivers.
Sandra Silvestri, MD, PhD, Chief Medical Officer of Ipsen (the company behind Bylvay), highlighted the transformative potential of this once-daily oral therapy for families living with PFIC’s challenges.
Global and Local Collaboration
The Japanese approval was achieved through a strategic collaboration between Ipsen and Jadeite Medicines Inc., with Jadeite leading the local clinical development and Ipsen responsible for commercialization.
Conclusion
The approval of Bylvay in Japan marks a turning point in PFIC care, providing a long-awaited, effective, and non-invasive treatment for a rare and life-threatening pediatric liver disease. For PFIC patients and their families, Bylvay offers renewed hope for improved quality of life and disease management.
